Myelodysplastic Syndromes (MDS)

A Phase Ib/II study of ceralasertib, a selective inhibitor of ATR, in patients with relapsed or refractory MDS and CMML

Pre-mRNA splicing gene mutations are common in MDS and CMML and induce R-loops which trigger ATR activation. We studied ceralasertib, an orally bioavailable ATR inhibitor, in adult patients with R/R MDS or CMML in a phase Ib/II study including a safety run-in and expansion of 160mg BID during a 28-day cycle on two schedules: days 1-14 (14on/14off) or days 1-7 and 15- 21 (7on/7off). Response rates and survival were estimated. Forty-four evaluable patients were treated.

Onyee Chan, MD

Institution
Moffit Cancer Center
Primary Disease Area of Focus
Acute Myeloid Leukemia (AML)
Aplastic Anemia
Chronic Myelomonocytic Leukaemia (CMML)
Myelodysplastic Syndromes (MDS)
Myeloproliferative Neoplasms (MPN)
About

Acute Myeloid Leukemia (AML), Chronic Myeloid Leukemia (CML), Chronic Myelomonocytic Leukemia (CMML), Leukemia, Myelodysplastic Syndromes (MDS), Myelofibrosis, Myeloproliferative Neoplasms (MPNs) Dr. Chan received her MD degree from the University of Arizona College of Medicine, Phoenix. She completed an Internal Medicine Residency at the University of Arizona College of Medicine in Tucson, followed by a Hematology/Oncology Fellowship at Moffitt Cancer Center/University of South Florida. Dr. Chan’s clinical interests include myeloid malignancies, specifically myelodysplastic syndrome (MDS) and

Teen Caregiver Finds New Purpose: Lilly's Story

“I would like other families to know that they are incredibly inspiring and strong. You are the foundations for patients with MDS to fight, you are their strength, their heart and their cheerleaders. Even when you're hurting, you show up for them, and you deserve to be recognized for the love you have for your family member that is fighting MDS. Never forget how important you are in this process.”

Myelodysplastic Syndromes in VEXAS 

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VEXAS syndrome is a clonal hemato-inflammatory disorder impacting older, predominantly male patients, characterized by systemic inflammation and progressive bone marrow failure. It is caused by somatic mutations in UBA1, an X-linked gene, essential for initiating cellular…

Donor cell-derived haematological neoplasms after allogeneic haematopoietic cell transplantation: recommendations from the EBMT Practice Harmonisation and Guidelines Committee 

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Summary Donor cell-derived haematological neoplasms (DDHN) are rare disorders and currently do not have standardised diagnostic criteria and therapeutic management. International experts in allogeneic transplantation and haematological malignancies from Europe, the Americas, and…

TP53 Mutation Acquisition Timing Influences Prognosis in MPNs, Study Finds

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The timing of TP53 mutation acquisition carries important prognostic implications for patients with myeloproliferative neoplasms (MPNs) and myelodysplastic syndromes/MPN overlap disorders. Later emergence of the mutation is associated with significantly worse survival outcomes…

Cancer Survivorship Is a Process, Not an Ending

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A blood cancer survivor debunks the movie myth of a quick cure, exposing the harsh, lasting side effects that remain after ringing the bell. One phrase I get tired of hearing lately is that something is a process. This word is often over-used and applies to anything from life…

Clinical outcomes of venetoclax combined with hypomethylating agents versus hypomethylating agents alone in TP53-mutated myelodysplastic syndromes 

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TP53-mutated (TP53-mt) myelodysplastic syndromes (MDS) are aggressive myeloid neoplasms associated with inferior responses to conventional chemotherapy and a high risk of progression to acute myeloid leukemia (AML).1-3 Although multiple targeted and immunotherapeutic strategies…

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